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JoVE Journal
Medicine

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Multi-exon Skipping Brug Cocktail antisenseoligonukleotider i Canine X-bundet Muskelsvindfonden
 
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Multi-exon Skipping Brug Cocktail antisenseoligonukleotider i Canine X-bundet Muskelsvindfonden

Article DOI: 10.3791/53776
May 24th, 2016

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Summary May 24th, 2016

Please note that all translations are automatically generated.

Click here for the English version.

Exon skipping er i øjeblikket en mest lovende behandlingsmulighed for Duchennes muskeldystrofi (DMD). For at udvide anvendeligheden for DMD patienter og optimere stabiliteten / funktion af de resulterende trunkerede dystrofin proteiner, blev en multi-exon springe tilgang med cocktail antisense oligonukleotider udviklet og vi viste systemisk dystrofin redning i en hund model.

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